National MPS Society

📢 Exciting News for the MPS IIIA Community! Ultragenyx has announced plans to expand the Transpher A clinical trial for UX111 (formerly ABO-102) by introducing a fourth Cohort! This cohort aims to provide additional insights into the investigational gene therapy. Learn more at clinicaltrials.gov. The sites will begin engaging with caregivers on January 13, 2025. Please respect this timeline as they finalize preparations. Caregivers interested in learning more should consult their child’s physician or contact the trial sites starting on January 13. We are so grateful for Ultragenyx's commitment to families affected by Sanfilippo syndrome. Thank you for inspiring hope for a brighter future. #MPS #RareDisease #Sanfilippo #MPSIII

We had so much fun with you all over the last couple of days... Mark your calendars for when we do it all again next year at the 39th Annual Family & Scientific Conference in Denver, Colorado! 🎉🥳 #NationalMPSSociety #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Time is running out— act now to protect critical legislation like the Creating Hope Reauthorization Act (H.R. 7384/S. 4583), Pediatric Priority Review Voucher (H.R. 5262/S. 1851), RARE Act (H.R. 7383/S. 1214), and more. It only takes two minutes to submit your statement on the National Organization for Rare Disorders, Inc. (NORD)'s website: https://ow.ly/8XVG50Uu3Lp #NationalMPSSociety #50Years #DisneyWorld #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

We’re excited to attend the National MPS Society’s Annual Family Conference this week in Orlando, FL, to honor and celebrate the organization’s 50 years of support, strength, and success for MPS families. REGENXBIO’s CEO, Curran Simpson, will join industry leaders for the CEO Fireside Chat and Dawn Phillips will share an update on our RGX-121 clinical program for the treatment of MPS II. We look forward to seeing the MPS community there!

The end of this year’s Congressional session is right around the corner! We need YOUR help advocating for several life-saving #RareDisease policy proposals. Urge your lawmaker to stand up for the rare disease community! ➡️ https://bit.ly/3VvveBy

🌟✨ Celebrate, Connect, and Discover: Join Us at the 38th Annual Family & Scientific Conference! ✨🌟 December 19-21, 2024, the National MPS Society is bringing the MPS and ML community together in Orlando, Florida, to celebrate 50 years of hope and progress! Our 38th Annual Family & Scientific Conference will feature cutting-edge research, meaningful connections, and opportunities to honor the incredible milestones we’ve achieved together. This year, we’re adding a touch of magic! While you engage in impactful sessions and connect with others, you'll also have the chance to make memories at nearby Walt Disney World. 💼 Whether you’re a researcher, family member, advocate, or supporter, this is your chance to make an impact and be inspired. 🌐 Register now: https://ow.ly/8Svn50UcZR5 ✨ Check out our free Disney tips: https://ow.ly/e0C850UcZOp Whether you’re coming for the science, the connections, or the celebrations, this is one conference you won’t want to miss. Let’s make magic together in Orlando! 🏰💜 #NMPSSC24 #NationalMPSSociety #50Years #DisneyWorld #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Joan Eppehimer, aunt to board member Jason Madison, recently joined the Forrager podcast to discuss her popcorn business-- and, along the way, she spread the word about #MPS and the Society! Check it out: https://ow.ly/t3Pv50UaQ1L #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

📢 The RDCRN Diversity Committee needs your help! They’re seeking individuals from rare disease communities—patients and caregivers—to participate in a study aimed at understanding barriers that prevent underrepresented racial and ethnic populations from joining rare disease clinical trials and research studies. 🔍 Details: A series of three focus groups will be held from December 11, 2024, through February 25, 2025. Learn more and get involved: https://ow.ly/ZtTJ50Uaef7 Please help spread the word by sharing the recruitment flyer across your networks— together, we can help make rare disease research more inclusive and representative. 💜 #RareDiseases #DiversityInResearch #PatientVoices

Over the weekend, staff and Society members had the pleasure of attending the grand opening of the Muenzer MPS Research & Treatment Center in Chapel Hill, North Carolina. Dr. Muenzer has had a profound and lasting impact on the MPS community through his dedication to research, patient care, and advocacy. We are so grateful for his efforts, his commitment to our community, and for the wonderful, concrete reminder of his legacy that is the new treatment center #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome

Since 2012, the FDA has awarded Priority Review Vouchers (PRVs) to companies developing life-saving and innovative therapies for children with rare diseases. This incentive has been instrumental in advancing treatments for conditions like MPS, ML, and related disorders. The Creating Hope Reauthorization Act (S. 4583) aims to extend the Pediatric Rare Disease Priority Review Voucher program through 2030. However, for this critical program to continue driving therapeutic breakthroughs, Congress must pass this legislation before the December 20, 2024 deadline. Help us advocate for the continuation of this legislation by sending a message directly to your Senators using this quick, easy tool from National Organization for Rare Disorders, Inc. (NORD): https://ow.ly/5n1u50TUBRO #MPS #ML #RareDisease #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome